Orphan Drugs Market: By Drug class (Biologics and Small Molecules) By Disease Type (Oncological Diseases, Metabolic Diseases, Hematologic and Immunologic Diseases, Infectious Diseases, and Neurologic Diseases) By Distribution channel (Hospitals Pharmacies, Retail Pharmacies, Online stores) Geography. 

Purchase Option

$ 4400
$ 6600
$ 8900

The orphan drugs market was valued at USD 174.11 million in 2023 and is poised to grow at a CAGR of 9.5% from 2024 to 2030. Orphan drugs are developed to treat rare medical conditions affecting less than 1 in 2,000 populations. Orphan drugs are used to treat patients with chronic severe diseases for which no treatment, or at least adequate one, has so far been available such as Agammaglobulinemia, Good pasture syndrome, Granulomatosis with Polyangiitis (GPA formerly Wagener Granulomatosis), Leucocyte adhesion deficiency, Pediatric Burton Agammaglobulinemia, Pediatric service combined Immunodeficiency, Schnitzler Syndrome and others. The orphan drugs market is growing significantly due to the rise in patient population, favorable reimbursement policies in developed and developing countries, and an increase in R&D for the launch of newer therapeutics by the market players. Moreover, pharmaceutical companies can price orphan drugs at premium levels due to the small patient population and the specialized nature of rare diseases, creating a proficient revenue potential.

In addition, regulatory incentives like decreased regulatory fees and tax credits further propel companies to invest in orphan drug development. Additionally, the rise in research and development efforts focused on addressing unmet medical needs, collaborations between academic institutions and market players, and fundraising from public and private organizations are anticipated to propel the orphan drugs market over the forecast years. However, the high cost of treatment, the stringent regulatory framework for product approval, and high R&D expenditure remain substantial barriers that hamper the growth of the orphan drugs market. However, the key opportunities associated with these drugs are enhanced affordability and patient access to these critical treatments, geographical expansion by the market players, and government incentives for R&D activities, which create opportunities for the players in the orphan drugs market.

Orphan Drugs Market Recent Key Developments:
  • In March 2023, ADX-2191 molecule developed by Aldeyra Therapeutics, Inc received First FDA-Approved Drug for Patients Suffering from Primary Vitreoretinal Lymphoma.
  • In September 2023, Ojjaara (momelotinib) received FDA approval for the myelofibrosis patients with anaemia condition.

Orphan Drugs Market Summary

Study Period

2024-2030

Base Year

2023

CAGR

9.5%

Largest Market

North America

Fastest Growing Market

Asia Pacific
Orphan Drugs Market Dynamics

The increasing prevalence of rare diseases in developed and emerging countries is a key factor in the surge of the orphan drugs market over the forecast years. For instance, Governments in developing countries are also implementing favorable policies, like India’s National police for rare diseases, providing a conducive environment for orphan drug manufacturers and market stakeholders. For instance, a recent study states that, in the United States, between 6,000 and 8,000 diseases have rare diseases, and it is affecting close to 25 million. Regardless of the huge number of individuals affected by rare diseases, pharmaceutical companies have been reticent to focus their efforts on drug development for these conditions due to not being able to recover the development costs.

Key Features of the Reports

  • The orphan drugs market report provides granular level information about the market size, regional market share, historic market (2019-2023) and forecast (2024-2030)
  • The report covers the in-detail insights about the competitor’s overview, company share analysis, key market developments and key strategies.
  • The report outlines drivers, restraints, unmet needs, and trends that are currently affecting the market.
  • The report track recent innovations, key developments and start-up details that are actively working in the market
  • The report provides a plethora of information about market entry strategies, regulatory frame work and reimbursement scenario.

Orphan Drugs Market Segmentation

By Drug Type
  • Biologics
  • Small Molecules
By Disease type
  • Oncologic Diseases
  • Metabolic Diseases
  • Hematologic and Immunologic Diseases
  • Infectious Diseases
  • Neurologic Diseases
  • Other rare diseases
By Distribution Channel
  • Hospitals Pharmacies
  • Retail Pharmacies
  • Online stores
Geography
  • North America
  • Asia Pacific
  • Latin America
  • MEA
  • Europe

Frequently Asked Questions

2021 is the base year and 2028 is the forecast year.

The report covers the five regions and 15+ countries market data: North America (United States, Canada), Europe (Germany, France, Italy, Spain, and United Kingdom (UK), Asia Pacific (China, India, Japan, Australia & New Zealand), Latin America (Brazil, Mexico, Argentina) and Middle East and Africa (Saudi Arabia, United Arab Emirates, South Africa).

In our report, we provide 12-15 market players’ information into the report. However, based on the client’s request we will provide additional country and regional market players information as well.

1.Executive Summary
2.Global Orphan Drugs Market Introduction 
2.1.Global Orphan Drugs Market  - Taxonomy
2.2.Global Orphan Drugs Market  - Definitions
2.2.1.Drug Type
2.2.2.Disease type
2.2.3.Distribution Channel
2.2.4.Region
3.Global Orphan Drugs Market Dynamics
3.1. Drivers
3.2. Restraints
3.3. Opportunities/Unmet Needs of the Market
3.4. Trends
3.5. Product Landscape
3.6. New Product Launches
3.7. Impact of COVID 19 on Market
4.Global Orphan Drugs Market Analysis, 2019 - 2023 and Forecast 2024 - 2030
4.1.  Market Analysis, 2019 - 2023 and Forecast, 2024 - 2030, (Sales Value USD Million)
4.2.  Year-Over-Year (Y-o-Y) Growth Analysis (%) 
4.3.  Market Opportunity Analysis 
5.Global Orphan Drugs Market  By Drug Type, 2019 - 2023 and Forecast 2024 - 2030 (Sales Value USD Million)
5.1. Biologics
5.1.1. Market Analysis, 2019 - 2023 and Forecast, 2024 - 2030, (Sales Value USD Million)
5.1.2. Year-Over-Year (Y-o-Y) Growth Analysis (%) and Market Share Analysis (%) 
5.1.3. Market Opportunity Analysis 
5.2. Small Molecules
5.2.1. Market Analysis, 2019 - 2023 and Forecast, 2024 - 2030, (Sales Value USD Million)
5.2.2. Year-Over-Year (Y-o-Y) Growth Analysis (%) and Market Share Analysis (%) 
5.2.3. Market Opportunity Analysis 
6.Global Orphan Drugs Market  By Disease type, 2019 - 2023 and Forecast 2024 - 2030 (Sales Value USD Million)
6.1. Oncologic Diseases
6.1.1. Market Analysis, 2019 - 2023 and Forecast, 2024 - 2030, (Sales Value USD Million)
6.1.2. Year-Over-Year (Y-o-Y) Growth Analysis (%) and Market Share Analysis (%) 
6.1.3. Market Opportunity Analysis 
6.2. Metabolic Diseases
6.2.1. Market Analysis, 2019 - 2023 and Forecast, 2024 - 2030, (Sales Value USD Million)
6.2.2. Year-Over-Year (Y-o-Y) Growth Analysis (%) and Market Share Analysis (%) 
6.2.3. Market Opportunity Analysis 
6.3. Hematologic and Immunologic Diseases
6.3.1. Market Analysis, 2019 - 2023 and Forecast, 2024 - 2030, (Sales Value USD Million)
6.3.2. Year-Over-Year (Y-o-Y) Growth Analysis (%) and Market Share Analysis (%) 
6.3.3. Market Opportunity Analysis 
6.4. Infectious Diseases
6.4.1. Market Analysis, 2019 - 2023 and Forecast, 2024 - 2030, (Sales Value USD Million)
6.4.2. Year-Over-Year (Y-o-Y) Growth Analysis (%) and Market Share Analysis (%) 
6.4.3. Market Opportunity Analysis 
6.5. Neurologic Diseases
6.5.1. Market Analysis, 2019 - 2023 and Forecast, 2024 - 2030, (Sales Value USD Million)
6.5.2. Year-Over-Year (Y-o-Y) Growth Analysis (%) and Market Share Analysis (%) 
6.5.3. Market Opportunity Analysis 
6.6. Other rare diseases
6.6.1. Market Analysis, 2019 - 2023 and Forecast, 2024 - 2030, (Sales Value USD Million)
6.6.2. Year-Over-Year (Y-o-Y) Growth Analysis (%) and Market Share Analysis (%) 
6.6.3. Market Opportunity Analysis 
7.Global Orphan Drugs Market  By Distribution Channel, 2019 - 2023 and Forecast 2024 - 2030 (Sales Value USD Million)
7.1. Hospitals Pharmacies
7.1.1. Market Analysis, 2019 - 2023 and Forecast, 2024 - 2030, (Sales Value USD Million)
7.1.2. Year-Over-Year (Y-o-Y) Growth Analysis (%) and Market Share Analysis (%) 
7.1.3. Market Opportunity Analysis 
7.2. Retail Pharmacies
7.2.1. Market Analysis, 2019 - 2023 and Forecast, 2024 - 2030, (Sales Value USD Million)
7.2.2. Year-Over-Year (Y-o-Y) Growth Analysis (%) and Market Share Analysis (%) 
7.2.3. Market Opportunity Analysis 
7.3. Online stores
7.3.1. Market Analysis, 2019 - 2023 and Forecast, 2024 - 2030, (Sales Value USD Million)
7.3.2. Year-Over-Year (Y-o-Y) Growth Analysis (%) and Market Share Analysis (%) 
7.3.3. Market Opportunity Analysis 
8.Global Orphan Drugs Market  By Region, 2019 - 2023 and Forecast 2024 - 2030 (Sales Value USD Million)
8.1. North America
8.1.1. Market Analysis, 2019 - 2023 and Forecast, 2024 - 2030, (Sales Value USD Million)
8.1.2. Year-Over-Year (Y-o-Y) Growth Analysis (%) and Market Share Analysis (%) 
8.1.3. Market Opportunity Analysis 
8.2. Europe
8.2.1. Market Analysis, 2019 - 2023 and Forecast, 2024 - 2030, (Sales Value USD Million)
8.2.2. Year-Over-Year (Y-o-Y) Growth Analysis (%) and Market Share Analysis (%) 
8.2.3. Market Opportunity Analysis 
8.3. Asia Pacific (APAC)
8.3.1. Market Analysis, 2019 - 2023 and Forecast, 2024 - 2030, (Sales Value USD Million)
8.3.2. Year-Over-Year (Y-o-Y) Growth Analysis (%) and Market Share Analysis (%) 
8.3.3. Market Opportunity Analysis 
8.4. Middle East and Africa (MEA)
8.4.1. Market Analysis, 2019 - 2023 and Forecast, 2024 - 2030, (Sales Value USD Million)
8.4.2. Year-Over-Year (Y-o-Y) Growth Analysis (%) and Market Share Analysis (%) 
8.4.3. Market Opportunity Analysis 
8.5. Latin America
8.5.1. Market Analysis, 2019 - 2023 and Forecast, 2024 - 2030, (Sales Value USD Million)
8.5.2. Year-Over-Year (Y-o-Y) Growth Analysis (%) and Market Share Analysis (%) 
8.5.3. Market Opportunity Analysis 
9.North America Orphan Drugs Market ,2019 - 2023 and Forecast 2024 - 2030 (Sales Value USD Million)
9.1. Drug Type Analysis 2019 - 2023 and Forecast 2024 - 2030 by Sales Value USD Million, Y-o-Y Growth (%), and Market Share (%) 
9.1.1.Biologics
9.1.2.Small Molecules
9.2.  Disease type Analysis 2019 - 2023 and Forecast 2024 - 2030 by Sales Value USD Million, Y-o-Y Growth (%), and Market Share (%) 
9.2.1.Oncologic Diseases
9.2.2.Metabolic Diseases
9.2.3.Hematologic and Immunologic Diseases
9.2.4.Infectious Diseases
9.2.5.Neurologic Diseases
9.2.6.Other rare diseases
9.3.  Distribution Channel Analysis 2019 - 2023 and Forecast 2024 - 2030 by Sales Value USD Million, Y-o-Y Growth (%), and Market Share (%) 
9.3.1.Hospitals Pharmacies
9.3.2.Retail Pharmacies
9.3.3.Online stores
9.4.  Country Analysis 2019 - 2023 and Forecast 2024 - 2030 by Sales Value USD Million, Y-o-Y Growth (%), and Market Share (%) 
9.4.1.United States of America (USA)
9.4.2.Canada
10.Europe Orphan Drugs Market ,2019 - 2023 and Forecast 2024 - 2030 (Sales Value USD Million)
10.1. Drug Type Analysis  and Forecast  by Sales Value USD Million, Y-o-Y Growth (%), and Market Share (%) 
10.1.1.Biologics
10.1.2.Small Molecules
10.2.  Disease type Analysis 2019 - 2023 and Forecast 2024 - 2030 by Sales Value USD Million, Y-o-Y Growth (%), and Market Share (%) 
10.2.1.Oncologic Diseases
10.2.2.Metabolic Diseases
10.2.3.Hematologic and Immunologic Diseases
10.2.4.Infectious Diseases
10.2.5.Neurologic Diseases
10.2.6.Other rare diseases
10.3.  Distribution Channel Analysis 2019 - 2023 and Forecast 2024 - 2030 by Sales Value USD Million, Y-o-Y Growth (%), and Market Share (%) 
10.3.1.Hospitals Pharmacies
10.3.2.Retail Pharmacies
10.3.3.Online stores
10.4.  Country Analysis 2019 - 2023 and Forecast 2024 - 2030 by Sales Value USD Million, Y-o-Y Growth (%), and Market Share (%) 
10.4.1.Germany
10.4.2.France
10.4.3.Italy
10.4.4.United Kingdom (UK)
10.4.5.Spain
11.Asia Pacific (APAC) Orphan Drugs Market ,2019 - 2023 and Forecast 2024 - 2030 (Sales Value USD Million)
11.1. Drug Type Analysis  and Forecast  by Sales Value USD Million, Y-o-Y Growth (%), and Market Share (%) 
11.1.1.Biologics
11.1.2.Small Molecules
11.2.  Disease type Analysis 2019 - 2023 and Forecast 2024 - 2030 by Sales Value USD Million, Y-o-Y Growth (%), and Market Share (%) 
11.2.1.Oncologic Diseases
11.2.2.Metabolic Diseases
11.2.3.Hematologic and Immunologic Diseases
11.2.4.Infectious Diseases
11.2.5.Neurologic Diseases
11.2.6.Other rare diseases
11.3.  Distribution Channel Analysis 2019 - 2023 and Forecast 2024 - 2030 by Sales Value USD Million, Y-o-Y Growth (%), and Market Share (%) 
11.3.1.Hospitals Pharmacies
11.3.2.Retail Pharmacies
11.3.3.Online stores
11.4.  Country Analysis 2019 - 2023 and Forecast 2024 - 2030 by Sales Value USD Million, Y-o-Y Growth (%), and Market Share (%) 
11.4.1.China
11.4.2.India
11.4.3.Australia and New Zealand (ANZ)
11.4.4.Japan
11.4.5.Rest of APAC
12.Middle East and Africa (MEA) Orphan Drugs Market ,2019 - 2023 and Forecast 2024 - 2030 (Sales Value USD Million)
12.1. Drug Type Analysis  and Forecast  by Sales Value USD Million, Y-o-Y Growth (%), and Market Share (%) 
12.1.1.Biologics
12.1.2.Small Molecules
12.2.  Disease type Analysis 2019 - 2023 and Forecast 2024 - 2030 by Sales Value USD Million, Y-o-Y Growth (%), and Market Share (%) 
12.2.1.Oncologic Diseases
12.2.2.Metabolic Diseases
12.2.3.Hematologic and Immunologic Diseases
12.2.4.Infectious Diseases
12.2.5.Neurologic Diseases
12.2.6.Other rare diseases
12.3.  Distribution Channel Analysis 2019 - 2023 and Forecast 2024 - 2030 by Sales Value USD Million, Y-o-Y Growth (%), and Market Share (%) 
12.3.1.Hospitals Pharmacies
12.3.2.Retail Pharmacies
12.3.3.Online stores
12.4.  Country Analysis 2019 - 2023 and Forecast 2024 - 2030 by Sales Value USD Million, Y-o-Y Growth (%), and Market Share (%) 
12.4.1.GCC Countries
12.4.2.South Africa
12.4.3.Rest of MEA
13.Latin America Orphan Drugs Market ,2019 - 2023 and Forecast 2024 - 2030 (Sales Value USD Million)
13.1. Drug Type Analysis  and Forecast  by Sales Value USD Million, Y-o-Y Growth (%), and Market Share (%) 
13.1.1.Biologics
13.1.2.Small Molecules
13.2.  Disease type Analysis 2019 - 2023 and Forecast 2024 - 2030 by Sales Value USD Million, Y-o-Y Growth (%), and Market Share (%) 
13.2.1.Oncologic Diseases
13.2.2.Metabolic Diseases
13.2.3.Hematologic and Immunologic Diseases
13.2.4.Infectious Diseases
13.2.5.Neurologic Diseases
13.2.6.Other rare diseases
13.3.  Distribution Channel Analysis 2019 - 2023 and Forecast 2024 - 2030 by Sales Value USD Million, Y-o-Y Growth (%), and Market Share (%) 
13.3.1.Hospitals Pharmacies
13.3.2.Retail Pharmacies
13.3.3.Online stores
13.4.  Country Analysis 2019 - 2023 and Forecast 2024 - 2030 by Sales Value USD Million, Y-o-Y Growth (%), and Market Share (%) 
13.4.1.Brazil
13.4.2.Mexico
13.4.3.Rest of LA
14. Competition Landscape
14.1.  Market Player Profiles (Introduction, Brand/Product Sales, Financial Analysis, Product Offerings, Key Developments, Collaborations, M & A, Strategies, and SWOT Analysis) 
14.2.1.Astrazeneca Plc
14.2.2.AOP Orphan Pharmaceuticals GmbH
14.2.3.Eli Lilly And Co.
14.2.4.Hoffmann-La Roche Ltd.
14.2.5.Gilead Sciences Inc.
14.2.6.Johnson & Johnson
14.2.7.Merck & Co.
14.2.8.Novartis Ag
14.2.9.Pfizer Inc.
14.2.10.Sanofi-Aventis
14.2.11.Teva Pharmaceutical Industries Ltd.
15. Research Methodology 
16. Appendix and Abbreviations 
  • Astrazeneca Plc
  • AOP Orphan Pharmaceuticals GmbH
  • Eli Lilly And Co.
  • Hoffmann-La Roche Ltd.
  • Gilead Sciences Inc.
  • Johnson & Johnson
  • Merck & Co.
  • Novartis Ag
  • Pfizer Inc.
  • Sanofi-Aventis
  • Teva Pharmaceutical Industries Ltd.

Adjacent Markets